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National Health Council Voluntary Health Leadership Conference

Margaret A. Hamburg MDFebruary 15, 2013Ponte Vedra Beach, Florida

Thank you. It is an honor to be speaking at your leadership conference. This is an impressive and influential group. I have enormous admiration for the work of the National Health Council, which so effectively brings together diverse stakeholders within the healthcare community to address the needs of people with chronic diseases and disabilities.

And this is an extraordinary moment in the public health, medical care and biomedical research...a time when, together, we are redefining the role—and the power—of patient advocacy organizations and the voices of the patients they represent.

Certainly we have come a long way from those days when patients were simply ignored or patronized by doctors who thought the patients’ views didn’t matter. And a long way from the early AIDS activists who initially had limited opportunities for making their concerns heard. But they insisted on doing so, and in the process achieved major change.

This is another such moment for change…and we see it happening…and we see that it matters.

Today, thanks in part to the success of its early beginnings, patient activism is taking on a whole new meaning...with an emphasis on “activism.” You now have expanded your advocacy voice, and became integral participants in the biomedical ecosystem, helping to guide medical product development and use across the product’s life cycle.

Today, patient organizations are funding academic research, supporting public-private partnerships, providing seed money for venture start-ups, identifying and recruiting clinical trial participants, supporting research with natural history data, aiding in the collection of postmarket data, and helping to support and train the next generation of scientists.

And, of course, almost all of you are active on the Hill—helping to shape important disease-specific legislation, like the Hope for Alzheimer’s Act, or broader legislative proposals like the MODDERN Cures Act, to help address significant unmet medical needs.

And NHC – and the organizations you represent—played such a critical role in shaping and securing the passage last year of the FDA Safety and Innovation Act (or FDASIA), which included the reauthorization of drug user fees, and established many other important new programs and initiatives.

This landmark law expanded the patient’s voice and demonstrated why your organization, which boasts of so many of the nation’s foremost patient advocacy groups, leads the efforts to make sure that policies and programs “put patients first.”

In addition, yours has been a clear voice about the importance of science as the driver of decision-making, and the need for adequate and appropriate investing in the full range of necessary research—including novel ideas and approaches—to ensure that patients and care givers get what they need.

And as we move towards personalized medicine, we’ll likely to see further changes in the role of patient organizations such as this one.

Amidst these tectonic changes for patient groups, my message for you today is two-fold. First, FDA firmly believes your voice matters. You bring a unique and important perspective to medical product development and innovation. We welcome your active role in the biomedical ecosystem and we will do what we can to further encourage and support that.

Secondly, I can assure you that as a public health agency, we at FDA never forget that we are working for patients. FDA is committed to finding new and efficient ways to get products to patients while ensuring those products are effective and used as safely as possible - and we want to partner with you in doing so.

This is very much a priority for the agency, and for me personally. I came to FDA convinced by decades of public health service, that the agency could be more of a catalyst for action -- that it could be more helpful in delivering on the promise of modern science and technology to address urgent unmet public health and medical care needs.

When I first met with you just over three years ago, I had only been in my job as FDA Commissioner for about 6 months. I was humbled then by the magnitude of the task, and I remain profoundly impressed by the challenges and opportunities we face at the FDA.

Let me take a moment to describe the vast public health agenda that FDA shoulders during this remarkable time; a time when unprecedented scientific advances – discoveries that frequently hold great therapeutic potential – coincide with critical gaps in our knowledge, tools and resources for translating this progress into promising medical products.

As you are no doubt aware, FDA is responsible for the safety, effectiveness, and quality of human drugs, vaccines and biologics, and medical devices, as well as animal drugs. We are responsible for the safety and quality of most of our nation’s food supply, the blood supply, and oversight of cosmetics, dietary supplements and certain products that emit radiation. In addition, since 2009 we’ve taken on the important task of regulating tobacco products.

And because the volume of imported food and medical products is increasing dramatically—quadrupling over the last decade-- we have come to recognize that to successfully protect U.S. public health, as well as to leverage the opportunities and insights of a scientific enterprise that knows no boundaries, we must think, act, and engage globally. Our interests must extend beyond our own borders.

That’s why we have opened 13 permanent offices in Asia, Europe, Latin America, Africa and the Middle East, and that’s why we are forming coalitions with our global regulatory partners, improving our capacity for data sharing and data analysis, and leveraging the efforts of public and private partners in all of our activities. We do have a lot to keep us busy.

One long-standing FDA initiative is our robust patient representative program. Now more than two decades old, this program was established because patients wanted and needed a more active role in medical product development.

This program brings the patient voice to the FDA advisory committee meetings to ensure its input into important medical product review and policy questions. Fittingly, the first Patient Representative served on the Antiviral Drugs Advisory Committee for HIV in 1991.

In more recent years, some of FDA review divisions have taken advantage of this program to seek the counsel of patient representatives at an early point in the product evaluation process outside the advisory committee meetings. Now, under FDASIA, we plan to expand that program to all of our drug review divisions and are currently at work on how to do that.

In part because of our experience with the patient representatives program and in part because of our recognition of the growing importance of patients in the biomedical ecosystem, FDA deliberately sought out your organization before the fifth reauthorization of PDUFA. We wanted to find out what really mattered to you -- to understand your perspectives and your priorities.

Armed with that information, our negotiators took the position that patient priorities would need to be a part of the final agreement…and industry negotiators agreed.

So PDUFA V really does represent a turning point for patients, as Marc Boutin has pointed out.

Consider the following: Among many things, you asked for and got a commitment to develop an objective, qualitative benefit-risk framework that includes robust patient input. You asked for -- and you got -- expanded use of biomarkers and patient-reported outcomes in clinical trials, as well as more funding for guidance, specialized training and agency outreach to the rare disease community.

I don’t need to tell you that patients who live with a disease have a direct stake in the outcome of drug review. At the same time, we believe the FDA drug review process would benefit from a more systematic and expansive approach to obtaining patients’ perspectives -- their views on disease severity, and the potential gaps or limitations in available treatments in a therapeutic area.

Because of that belief, FDA has been developing an enhanced, structured approach to benefit-risk assessments that accurately and concisely describes these considerations in the agency’s drug regulatory decision-making. Part of FDA’s decision-making lies in thinking about the context of the decision – an understanding of the condition to be treated and the unmet medical need.

PDUFA V expanded the implementation of this risk-benefit framework, which we honed at a public workshop last May. That framework will provide the big picture about a regulatory decision – as well as bottom-line descriptions of the issues involved in that decision.

It should make more explicit the issues involved in the review of a product and should foster discussions of what really matters when a regulatory decision is made. And by clearly articulating the clinical reasoning and judgment behind regulatory decisions, it can improve transparency.

Another important aspect of PDUFA V has to do with patient-reported outcomes. Assessments of study endpoints -- known as patient-reported outcomes -- are increasingly an important part of successful drug development. They help measure, from the patients’ point of view, treatment benefit or risk in clinical trials.

To help guide our benefit risk-assessments of new pharmaceutical products, PDUFA V supports the Patient Focused Drug Development Initiative. Its purpose is to learn from patients about the impact of their disease on their daily lives.

Our plan is to hold at least 20 public meetings over the next 5 years, each focused on a different disease area, and we expect that these gatherings will be attended not only by our staff and patient representatives, but also potential sponsors of new drug development.

Our first public meeting, held last October, was designed to help us identify which disease areas to focus on, and we will soon announce the final choices.

And you should know that the drug center isn’t the only FDA product center that is turning to patients for advice.

For example, the diabetes community actively engaged with FDA when our Center for Devices and Radiological Health (CDRH) was writing a guidance on the development of an artificial pancreas. It was extremely valuable. Now that center has launched the Patient Preference initiative, because it wants to find new ways to incorporate into its review process the views of patients on the potential risks and benefits of the products they’ll be using.

These are some of the kinds of new partnerships we need to help bring better, safer products to people -- whether it’s a senior citizen whose function is impaired by the devastation of Alzheimer’s disease, someone suffering from complications of heart disease, a child with poorly controlled type 1 diabetes, or a mother who has just learned that she has ovarian cancer.

Success will require a new openness, and regulatory flexibility in light of continually changing needs and circumstances. But it also demands that we advance regulatory science: the knowledge and tools necessary for the meaningful and timely development of new products and their review for safety, efficacy, quality and performance.

Unfortunately, this has been an area of our overall scientific investments and enterprise that has been woefully under-addressed. But it is an area that is absolutely vital if we, as a nation, want to leverage today’s opportunities in science in the form of new, better and more innovative medical products.

Though not everyone gets it, I know that you understand and support this important agenda of our agency. In fact, one of the very first times that I formally spoke about regulatory science to an outside group was to your organization.

Frankly, I was uncertain how my message would be received. I had been warned that as important as this area of science might be, it wasn’t sexy, discovery science. At best, people’s eyes would glaze over, but worse…anything with the word regulatory in its name would likely be met with an automatic aversive reaction in our current anti-regulatory climate.

I’ll admit that you all were, in a sense, my guinea pigs. But you looked and acted interested and engaged. And, in fact, you understood what it was and why it matters as the critical link between opportunities in science—those exciting, sexy discoveries—and the ability to translate them into real world products for people who need them.

Perhaps more than others, patient groups understand the pressing need to bridge the wide gap that often separates promising scientific discoveries from the development, testing and manufacture of new products that meet FDA’s standards for safety, effectiveness, performance and quality.

Your response encouraged me ...and for that I am grateful. But more importantly, you have been champions of the concept of broadening how we think about investments in research to be sure that we are able to leverage science more fully in the service of people.

And the patient community made a real and enduring difference when you recognized and spoke to the importance of regulatory science in the weeks and months leading up to the passage of FDASIA.

Advancing regulatory science must be a huge priority, not just within FDA, but as an active, dynamic field of scientific research.

We must continually work to find new and better ways of doing things…To seize new opportunities that exist in science and technology…And to work with industry and our scientific partners in academia and government…and with patients—in a collaborative way-- to discover and apply new regulatory tools… and to speed the development of new products.

Such things as: enhancing the use of pharmacogenomics and qualified biomarkers; developing innovative clinical study designs that enable clinical trials to be smaller, more efficient, and more adaptive; and developing new strategies using bioinformatics to more effectively mine large data bases to learn more about issues of both safety and effectiveness.

These efforts matter in our ability to swiftly and surely review product applications that come before us…and they are also essential for reducing the time and cost -- and increasing the likelihood of success -- for drug development itself.

And some striking progress has been made. Thanks to better science, new regulatory tools and approaches, and carefully developed products and applications, last year, FDA was able to approve 39 novel drugs (new molecular entities) – the highest total in 16 years. . drugs that will really benefits patients.

A large number of these new therapies are for people with urgent needs who suffer from chronic disease conditions.: new treatments for multiple sclerosis, chronic renal failure, COPD, HIV, rheumatoid arthritis, familial hypercholesterolemia Cushing’s disease, cystic fibrosis, several different cancers, and much more.

The new approvals included pathbreaking new treatment approaches and targeted therapies linking genetic markers to specific treatments in truly transformative ways.

Strikingly, FDA reviewed many of these new drugs quickly using its several review authorities for important new drugs, including Fast Track, Priority Review, and Accelerated Approval.

Moreover, strengthened communication with drug companies early in development and flexible clinical trial designs for drugs for unmet medical needs also enabled drug companies to conduct shorter, smaller, or fewer studies, reducing the length and cost of drug testing.

The upshot was that many of these important drugs were made available to patients in this country earlier than anywhere else in the world.

Also, I know that many NHC patient groups represent rare diseases. We are keenly aware that there are treatment options for only a small percentage of the 7,000 rare diseases that collectively affect over 30 million Americans.

But we take heart in the fact that one out of three new drug approvals in the past five years have been for rare diseases…but it wasn’t always that way.

The Orphan ‘Drug Act, which provides incentives and a path to market for drugs intended to treat patients with rare diseases, is now 30 years old.

In the decade leading up to the passage of this act, only 10 industry-supported products had been developed for rare diseases.

Since then, over 400 orphan products have been brought to the market. I think we can say that the program is working.

FDA is at work on other approaches that will also enhance drug innovation.

Importantly, we are currently implementing a provision in FDASIA that will further spur drug innovation – a new pathway for breakthrough therapies.

This breakthrough pathway is designed for when early scientific work suggests that a new drug holds real promise to offer substantial improvements over available therapies to treat a serious condition.

In addition to all of the features of fast track designation, a sponsor that receives a breakthrough designation for their drug will generally receive more intensive guidance on an efficient drug development program, beginning as early as phase 1, as well as the involvement of senior FDA management.

As of yesterday, FDA had received a total of 22 breakthrough therapy designation requests, of which five have been granted, five have been denied, and 12 are pending.

And we’re currently exploring yet another potential pathway for initial approval of promising drugs that show overall benefit to address an unmet medical need in a specific subgroup of patients. Such drugs would be approved under a designation of Special Medical Use, signaling strongly to payors and prescribers the limited population that should be prescribed a drug. The thinking is that such drugs might be approved based on smaller and shorter clinical trials for a more limited, specific group, but then approval could be broadened for use for lower risk individuals in a broad disease category as we learn more about safety and efficacy. Obesity is an example of an area where we think such an approach could have real value.

FDA’s center for devices also has exciting programs underway to encourage innovations. Just yesterday, CDRH approved the first retinal implant for adults with a rare genetic disease that damages the light sensitive cells that line the retina.

The Argus II Retinal Prosthesis System was approved as a humanitarian use device, an approval pathway limited to those devices that treat or diagnose fewer than 4,000 people in the United States each year. Data supporting approval was based on a clinical trial of 30 patients.

CDRH cares deeply about capturing the power of advancing science and technology, and has developed the Innovation Pathway to encourage breakthrough devices and diagnostic tests. The center is currently testing this model in a small group of products to treat end stage renal disease.

Of course, pathways and meetings aren’t the only way that FDA encourages medical product development. We’re also committed to issuing guidance documents that spell out our thinking. Just last week we announced a guidance for companies developing new treatments for patients in the early stages of Alzheimer’s disease, before the onset of noticeable dementia.

This guidance document explains the FDA’s current thinking about how to identify and select patients with early Alzheimer’s disease for participation in clinical trials… and I think it will make a real difference in this important and undeveloped area.

In 2011, we issued guidance to encourage the development of companion drug and diagnostics to help individual treatments, and we are moving toward issuing a final guidance document.

We know that there are enormous opportunities to improve health and prevent disease if we can truly leverage all that is happening in the world of science and research to address medical product needs throughout the lifecycle of a product, from initial discovery to the marketplace. We can make new treatments and cures available, along with new strategies to better diagnose and prevent disease.

Especially in these difficult budgetary times, we certainly appreciate you speaking out for continued funding of regulatory science and biomedical intervention. Leveraging resources and people and dollars is one way around the budget conundrum.

It is the hallmark of the Reagan-Udall Foundation and the new Medical Device Innovation Consortium and the other public-private partnerships that we are increasingly engaged in. These are exciting and valuable models for advancing science and understanding. I urge you to continue to explore and participate in such models. Certainly, together, we need to find more approaches and pursue more partnerships to speed up the cures, treatments and preventive measures that we all so fervently wish for…and that so many desperately need.

There is so much to talk about and time is passing ….but before I close, I would be remiss if I did not speak, at least briefly to the other side of the FDA house…food and nutrition. As you know, enormous progress can be made in the realm of chronic disease prevention and management, through sound programs and policies.

It is increasingly evident that unhealthy diets and physical inactivity are contributing to the burden of many chronic diseases and conditions, including type 2 diabetes, hypertension, heart disease, stroke, and some cancers; not to mention the alarming increases in overweight and obesity, among adults and children, with all its attendant health risks.

To address some of these concenrs, FDA is working to update nutrition labeling, including menu labeling, so that consumers have better access to useful and understandable nutrition information about the foods they purchase and consume.

We have been deeply engaged also in efforts to reduce sodium in the food supply, and we are committed to work with industry to make progress in this important domain. Significant progress has been made to bring down transfats in the American food supply, thanks in large part to labeling requirements—and it appears to be making a difference for health. But there is more progress to be made there as well.

And since the last time I spoke with you, we have made enormous progress in the implementation of the Tobacco Prevention and Control act, signed into law by President Obama in the summer of 2009. We cannot forget that tobacco use remains the number one cause of preventable death in the United States, and that it is responsible for an enormous amount of chronic disease and disability. With our new authority over tobacco products, we now have the potential to substantially reduce tobacco use, especially by young people, and help turn the tide on an epidemic of preventable disease and death. So let me close where I began…with thanks for the opportunity to be with you here today…and to reiterate my deep respect for you, this organization and the organizations you all represent. You are powerful partners in the struggle to promote health and prevent disease. I am truly grateful for your work and look forward to our continuing work together.